Real-world Treatment Patterns Research Articles

Real-world treatment patterns and quality of life among patients with locally advanced or metastatic urothelial carcinoma living in Saudi Arabia, South Korea, Taiwan, and Turkey.

To evaluate demographic and clinical characteristics, treatment patterns, and quality of life in patients with locally advanced or metastatic urothelial carcinoma in Asia. Data were drawn from the Adelphi Real World Metastatic Urothelial Carcinoma Disease Specific Programme™, a cross-sectional survey of medical oncologists/urologists and their adult patients in Saudi Arabia, South Korea, Taiwan, and Turkey. Exploratory patient-reported outcomes included the EQ-5D visual analog scale, European Organisation for Research and Treatment of Cancer Quality of Life of Patient Questionnaire global health, and Brief Pain Inventory. Analyses were descriptive. Overall, 175 physicians reported data for 988 patients. Mean (standard deviation) patient age was 66.3 (10.8) years, 77% were men, and 82% had bladder tumors at diagnosis. Of patients receiving first- (n = 988), second- (n = 290), and third-line (n = 87) treatments, 81%, 35%, and 59% received chemotherapy, respectively, and 17%, 63%, and 34% received programmed cell death protein 1/ligand 1 inhibitors, respectively. Patient-reported (n = 319) mean (standard deviation) EQ-5D visual analog scale score was 51.8 (15.6), European Organisation for Research and Treatment of Cancer Quality of Life of Patient Questionnaire global health status score was 44.6 (19.9), and Brief Pain Inventory score was 6.5 (1.9; n = 315). The most common first- and second-line treatments for locally advanced or metastatic urothelial carcinoma were chemotherapy and programmed cell death protein 1/ligand inhibitors, respectively. At third line, 10% of patients received best supportive care alone, underscoring an unmet need for effective third-line treatment options. Patients in all regions reported quality-of-life impairment.

Clinical characteristics, treatment patterns, and outcomes in adult patients with germline BRCA1/2-mutated, HER2-negative advanced breast cancer: a retrospective medical record review in the United States

AimTo examine clinical characteristics, real-world treatment patterns, and health outcomes among patients with germline BRCA1/2-mutated, human epidermal growth factor receptor 2 (HER2)–negative advanced breast cancer (ABC).MethodsA retrospective analysis was conducted using medical records from patients with HER2-negative ABC with BRCA1/2 mutation who received cytotoxic chemotherapy. Data were stratified into groups with triple-negative breast cancer (TNBC) or hormone receptor–positive (HR+)/HER2-negative diagnoses. Time-to-event outcomes (i.e., real-world progression-free survival [rwPFS] and overall survival [OS]) were calculated to summarize health outcomes.ResultsWhen diagnosed with ABC, most patients were younger than 60 years (mean age = 57.3 years), were white (76.4%), and had a family history of BRCA-related cancer (71.5%). A total of 305 patient records were examined; 194 patients (63.6%) had advanced TNBC, and 111 patients (36.4%) had HR+/HER2-negative ABC. Chemotherapy was primarily used as first-line treatment for both subgroups, but the TNBC subgroup received poly (ADP-ribose) polymerase (PARP) inhibitors at triple the rate as a second-line treatment and double the rate as a third-line treatment compared with the HR+/HER2-negative subgroup. Two-year OS rates were similar between the TNBC (73.9%) and the HR+/HER2-negative subgroups (77.0%), and anemia, nausea, and neutropenia were the most commonly reported toxicities across all treatments.ConclusionClinicians should consider the use of targeted agents such as PARP inhibitors in earlier lines of therapy for ABC given the growing evidence that PARP inhibitors may improve PFS compared with chemotherapy while potentially offering a more manageable toxicity profile and improved quality of life.

Real-World Treatment Patterns Among Patients with Type 2 Diabetes Mellitus Initiating Treatment with Oral Semaglutide.

The treatment landscape for type 2 diabetes mellitus (T2DM) is complex and constantly evolving, and real-world evidence of prescribing patterns is limited. The objectives of this study were to characterize lines of therapy (LOTs), calculate the length of time spent on each LOT, and identify the reasons for the LOT end among patients who initiated oral semaglutide for T2DM. This retrospective, claims-based study included commercial and Medicare Advantage adults with T2DM. Data from November 1, 2019, and June 30, 2020, were obtained from Optum Research Database. Patients with ≥ 1 claim for oral semaglutide and continuous health plan enrollment for ≥ 12months prior to (baseline period) and ≥ 6months following (follow-up period) the date of the first oral semaglutide claim were included. LOT 1 began on the date of the first oral semaglutide claim. The start date of any subsequent LOTs was the date of the first claim for an additional non-insulin anti-diabetic drug class or a reduction in drug class with use of commitment medications. The LOT ended at the first instance of medication class discontinuation, change in regimen or end of follow-up. Of the 1937 patients who initiated oral semaglutide, 950 (49.0%) remained on their initial regimen over the 6-month follow-up period, 844 (43.6%) had at least one subsequent LOT, and 89 (4.6%) had at least two subsequent LOTs. Among patients with more than one LOT, approximately 20%-25% used oral semaglutide as monotherapy or combination therapy during LOTs 2 and 3. Metformin was frequently used during treatment across all LOTs. This study provides insight for physicians and payers into the real-world prescribing practices within the first 6months following oral semaglutide initiation and fills the gap in understanding the frequency of regimen changes in the constantly evolving and complex environment of T2DM care.

Real-World Treatment Patterns, Clinical Outcomes, and Symptom Burden in Patients With Psoriatic Arthritis Prescribed Ixekizumab in the United States.

The objective of this study was to describe the real-world characteristics and clinical status of patients with psoriatic arthritis (PsA) currently prescribed ixekizumab. Data were drawn from the Adelphi PsA Plus Disease Specific Programme (DSP), a cross-sectional survey conducted in the United States between September 2021 and March 2022. Rheumatologists provided data for their next five consulting patients currently receiving ixekizumab, including demographic and clinical characteristics, disease severity, treatment history, reasons for treatment choice, satisfaction with current treatment, and current and historic symptom burden. Patients voluntarily completed questionnaires, providing perceptional data on symptom burden and satisfaction with current treatment. Overall, 68 rheumatologists provided data on 275 patients with PsA, 90 of whom completed the voluntary questionnaire. Patients had been prescribed ixekizumab for a mean of 11.7 (SD 10.6) months. Clinical characteristics, disease severity, and symptom burden of patients with PsA improved significantly from ixekizumab initiation to the most recent consultation, including symptom burden, tender and swollen joint counts, and body surface area affected by psoriasis (all P < 0.001). Both rheumatologists and patients were satisfied with ixekizumab treatment and reported improvements in pain and fatigue. Improvements were noted after more than three months of ixekizumab treatment duration and regardless of whether the patients had prior exposure to an advanced therapy or were treatment naïve. Our results indicate that ixekizumab was efficacious in the treatment of PsA in real-world clinical practice, complementing efficacy data from randomized controlled clinical trials. The results of this study may assist rheumatologists and their patients in making informed treatment choices.

Abstract PO2-17-06: Real world treatment patterns and clinical outcomes by germline BRCA (gBRCA) mutation status in patients with HER2-negative early breast cancer in the US community oncology setting: a retrospective observational chart-review study

Abstract Background: Women with early-stage breast cancer (eBC) who have inherited BRCA1 or BRCA2 mutations (gBRCAm) are typically diagnosed at a younger age and often require more intensive treatment. In the randomized, double-blind OlympiA trial of HER2-negative (HER2–) gBRCAm patients, olaparib, a targeted poly (ADP-ribose) polymerase inhibitor (PARPi), significantly improved invasive disease free survival (IDFS), distant disease free survival (DDFS) and overall survival (OS) and was subsequently approved for adjuvant treatment of gBRCAm HER2– high-risk eBC patients. This study examined the real-world patient characteristics, treatment patterns and clinical outcomes by gBRCA status among patients with HER2– eBC in a US community oncology setting prior to olaparib US approval for treatment of eBC. Methods: This retrospective observational study used chart review data from The US Oncology Network's iKnowMed database to examine adult patients diagnosed with HER2– eBC (stage I-III) initiating systemic neoadjuvant or adjuvant therapy with chemotherapy and/or endocrine therapy between January 1, 2012 and December 31, 2018. Patients with valid gBRCAm test results (all gBRCAm and randomly selected BRCAwt patients) were included and followed through December 31, 2021. Patients were excluded if they had HER2+ tumors and progressed to stage IV within 6 months after initiation of neoadjuvant therapy or were diagnosed with another primary cancer. Descriptive analyses assessed patient characteristics. Kaplan Meier methods and multivariate Cox proportional hazard models (CPHM) were used to evaluate IDFS, DDFS and OS by gBRCA status and by HR+/HER2– or TNBC eBC subsets. Results: Among 298 BC patients meeting initial criteria, 42% had gBRCAm (n=124, 43% hormone receptor positive [HR+], 56% triple-negative BC [TNBC], 1% unknown), and 58% had gBRCAwt (n=174, 74% HR+, 22% TNBC). Median (interquartile) age at surgery was numerically lower in gBRCAm (45 [36,55] years) than gBRCAwt (48 [41,55] years) patients. A numerically higher proportion of gBRCAm patients received neoadjuvant therapy compared with gBRCAwt (Neoadj: gBRCAm 37.1% vs gBRCAwt 12.6%) but the reverse was true for adjuvant (Adj: gBRCAm 42.7% vs gBRCAwt 65.5%) and neoadjuvant + adjuvant use was numerically similar (Neoadj+Adj: gBRCAm 20.1% vs gBRCAwt 21.8%). The majority of TNBC patients received neoadjuvant therapy while the majority of HR+ patients received adjuvant therapy (Neoadj: TNBC 51.4% vs HR+ 6.6%, Adj: TNBC 29.0% vs HR+ 71.4%, Neoadj+Adj: TNBC 19.6% vs HR+ 22.0%). Median IDFS, DDFS, and OS in both study cohorts were not reached. Median follow-up from surgery was &amp;lt; 5 years (overall 59.0 months; gBRCAm 50.6 months; gBRCAwt 61.3 months). At 60 months, in gBRCAm and gBRCAwt respectively, estimated IDFS were 85.5% and 90.9%, estimated DDFS 88.1% and 92.2%, and estimated survival 91.9% and 95.2%. At 60 months, in HR+/HER2– and TNBC respectively, estimated IDFS were 92.4% and 81.2%, estimated DDFS 93.9% and 85.7%, and estimated survival 96.3% and 90.9%. CPHM results showed that risks of invasive disease (HR 1.74; 95% CI 0.83-3.64; p=0.15), distant disease (HR 1.24; 95% CI 0.50-3.07; p=0.65) and death (HR 1.79; 95% CI 0.52-6.13; p=0.353) were similar between gBRCAm and gBRCAwt patients. Between HR+/HER2– and TNBC patients, risks of invasive disease (HR=0.63; 95% CI 0.21-1.89; p=0.41), distant disease (HR=0.42; 95% CI 0.11-1.67; p=0.22) and death (HR 0.48; 95% CI 0.09-2.70; p=0.41) were similar. Conclusions: This real-world chart review study of data through 2021 (before PARPi approval for eBC) with limited follow up of 5 years found that gBRCAm patients had a numerically shorter survival (IDFS, DDFS and OS) than gBRCAwt, but this difference was not statistically significantly different. Given that the follow-up period was too short to draw conclusions, further studies examining clinical outcomes over a longer follow-up period after approval of PARPi may be of interest. Citation Format: Jay Andersen, Jagadeswara Earla, Nicole Fulcher, Juliet Ndukum, Nicholas J Robert, Mark Robson, Weiyan Li, Jaime Mejia. Real world treatment patterns and clinical outcomes by germline BRCA (gBRCA) mutation status in patients with HER2-negative early breast cancer in the US community oncology setting: a retrospective observational chart-review study [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO2-17-06.

Abstract PO3-16-08: Real-World Treatment Patterns in Elderly Patients with HER2-Negative Early Breast Cancer

Abstract Background: As the treatment landscape of human epidermal growth factor receptor 2-negative (HER2–) early breast cancer (BC) evolves, an understanding of treatments given in real-world settings to elderly patients with HER2– early BC is needed to gain insight into the potential unmet needs of this population for whom clinical data are limited. This study described demographics, clinical characteristics, and neoadjuvant and adjuvant treatment patterns in elderly patients with HER2– early BC in the United States. Methods: This retrospective study used SEER-Medicare data (2010–2019) to identify patients aged ≥66 years with HER2– early (Stage I–III) BC receiving primary surgery. Patient characteristics were evaluated at primary surgery or at BC diagnosis. Neoadjuvant and adjuvant therapies were defined as any National Comprehensive Cancer Network-recommended regimens received between BC diagnosis and primary surgery, and during the 180-day period following primary surgery, respectively. Patient characteristics were described using means and standard deviations (SDs) for continuous characteristics, and frequencies and proportions for categorical characteristics. Neoadjuvant and adjuvant therapies were summarized using frequencies and proportions. All analyses were conducted for the overall population and stratified by breast cancer subtype (hormone receptor-positive [HR+]/HER2– vs. triple negative BC [TNBC]) and stage at diagnosis (Stage I vs. Stage II/III). Results: Of 28,655 eligible patients, 25,899 (90.4%) had HR+/HER2– BC and 2,756 (9.6%) had TNBC; 17,961 (62.7%) had Stage I BC and 10,694 (37.3%) had Stage II/III BC. Mean (SD) age at diagnosis was 75.8 (6.4) years, mean (SD) National Cancer Institute comorbidity index was 1.9 (2.1), and 23.7% received single or multi-gene testing for a breast cancer gene 1 and/or 2 (BRCA1/2) mutation (HR+/HER2– BC: 24.6%; TNBC: 15.5%), with most (86.3%) testing occurring after primary surgery. Relative to HR+/HER2– BC patients, TNBC patients were less likely to present with Stage I disease (46.7% vs. 64.4%), had a larger mean tumor size at diagnosis (2.6 cm vs. 1.9 cm), and higher-grade tumors (Grade III or IV: 70.6% vs. 14.6%). TNBC patients were also more likely to receive neoadjuvant chemotherapy (90.9% vs. 35.3%) and adjuvant radiation therapy (81.2% vs. 66.4%) than HR+/HER2– BC patients (Table 1). By BC stage, neoadjuvant therapy was more common among Stage II/III patients relative to Stage I patients (12.3% vs. 2.7%), with most (90.4%) neoadjuvant chemotherapy use observed among Stage II/III patients. Adjuvant therapy use was similar between Stage I and Stage II/III patients (87.4% vs. 88.1%) and the most common adjuvant therapies across both subgroups were endocrine therapy (85.2% vs. 82.2%) and radiation therapy (68.5% vs. 66.1%). Conclusion: In elderly patients with HER2– early BC, treatment with adjuvant therapy was common, while neoadjuvant therapy was limited. Moreover, one in four HER2– early BC patients were tested for a BRCA1/2 mutation in real-world clinical practice. Our findings highlight the need for a better understanding of the role of increased and timely BRCA1/2 testing to inform optimal treatment strategies across neoadjuvant and adjuvant settings in this population. Table 1. Treatment Patterns Among Patients with TNBC and HR+/HER2– Early Breast Cancer Citation Format: Jagadeswara Earla, Allison Kurian, Kalé Kponee-Shovein, Ambika Satija, Yan Song, Nathaniel Downes, Malena Mahendran, Qi Hua, Arun Kumar, Jaime Mejia. Real-World Treatment Patterns in Elderly Patients with HER2-Negative Early Breast Cancer [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO3-16-08.

Abstract PO1-05-05: Overall survival (OS) and subsequent therapy patterns in Japanese patients with HR+/HER2− advanced breast cancer (ABC) treated with palbociclib (PAL) plus letrozole (LET) in first-line setting (1L)

Abstract Background: PAL is a first-in-class cyclin-dependent kinase 4/6 inhibitor (CDK4/6i) for the treatment of HR+/HER2− ABC. In PALOMA-2 study, PAL plus LET demonstrated a numerically, not statistically significant, prolonged OS versus placebo plus LET (53.9 vs 51.2 months; HR, 0.956; 95% CI, 0.777–1.177) in patients with HR+/HER2− ABC in 1L. Subgroup analyses showed a similar trend in Asia/Pacific region (73.4 vs 55.1 months; HR, 0.744), however, data specific to Japanese subgroup have not been reported. An open-label, single-arm, multicenter Japanese phase 2 study (J-Ph2) investigated the efficacy and safety of PAL plus LET in postmenopausal Japanese women with HR+/HER2− ABC in 1L and showed a median progression-free survival (PFS) of 35.7 months (95% CI, 21.7–46.7) in the final analysis of PFS. However, OS data were immature at the data cutoff. Here, we report the findings from a follow-up study of J-Ph2 (NCT04735367) evaluating OS and subsequent therapy in these Japanese patients. Methods: Patients (N=42) who participated in J-Ph2 were enrolled in this follow-up study. The primary endpoint was OS, defined as the time from the first dose of PAL plus LET in J-Ph2 to date of death due to any cause. Secondary endpoints included chemotherapy-free survival (CFS) and type and duration of subsequent therapy. Median OS, CFS, duration of subsequent therapy and associated 95% CIs were estimated using the Kaplan–Meier method. Outcomes were also assessed for baseline demographic and disease characteristic subgroups including visceral or nonvisceral metastatic disease, disease-free interval (DFI; ≤12 months, &amp;gt;12 months, de novo metastatic), and duration of 1L PAL use (≤24 months or &amp;gt;24 months). Results: Patients had a median age of 62.5 years; 48% had visceral metastases; 33% had de novo disease; 48% had a TFI &amp;gt;12 months; 93% had an ECOG performance status (PS) of 0. At a median follow up of 89.7 months, the median OS was 85.4 months (95% CI, 64.3–not estimable [NE]). Median OS was longer in patients with nonvisceral versus visceral metastases (not reached [NR] vs 67.3 months), with TFI &amp;gt;12 months versus ≤12 months (85.4 vs 45.4 months), or with duration of 1L PAL use &amp;gt;24 months versus ≤24 months (NR vs 47.5 months). Median CFS was 69.1 months (95% CI, 24.2–85.4), and that was longer in patients with nonvisceral versus visceral metastases (77.5 vs 37.5 months). At the data cutoff, 3 patients (7.1%) were still receiving PAL plus LET (90.9–93.2 months). Subsequent therapy after disease progression was administered to 34 of 42 patients (81%). Of these patients, 28 (82%) received endocrine-based therapy, while 3 (9%) patients each received chemotherapy and other therapy, respectively. The median duration of the first subsequent therapy was 8.3 months (95% CI, 3.9–12.2), and that was similar among patients with nonvisceral versus visceral metastases (7.8 vs 8.3 months). Conclusion: This interim analysis showed a median OS of &amp;gt;7 years with 1L PAL plus LET. Patient demographics (geographic, racial factors), disease characteristics (ECOG PS, visceral metastases), and subsequent therapy decisions may have contributed to the extended median OS observed in this study. Further, this report provides insight into real-world subsequent treatment patterns following PAL plus ET. Table Citation Format: Masato Takahashi, Tomofumi Osako, Hiroyuki Yasojima, Kenichi Inoue, Masahiro Kawashima, Hideki Maeda, Mitsuya Ito, Yasuaki Sagara, Kan Yonemori, Masaya Hattori, Naohito Yamamoto, Eriko Tokunaga, Shozo Ohsumi, Yutaka Yamamoto, Akemi Ichikawa, Yasuaki Muramatsu, Norikazu Masuda. Overall survival (OS) and subsequent therapy patterns in Japanese patients with HR+/HER2− advanced breast cancer (ABC) treated with palbociclib (PAL) plus letrozole (LET) in first-line setting (1L) [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO1-05-05.

Brentuximab Vedotin Retreatment in Patients with Relapsed or Refractory Classical Hodgkin Lymphoma or Peripheral T-Cell Lymphoma: A Retrospective United States Claims Analysis.

Brentuximab vedotin (BV) monotherapy (BV-M) and combination (BV-C) therapies are safe and effective for classical Hodgkin lymphoma (cHL) and CD30-expressing peripheral T-cell lymphomas (PTCLs). Although the sample sizes have been small (12-29 patients), in clinical studies, response rates of 53-88% have been reported for BV retreatment in patients with an initial BV response. We evaluated the real-world characteristics and treatment patterns of cHL/PTCL patients who received BV and were retreated in the United States. Symphony Health Patient Claims (11/2013-1/2022) were retrospectively analyzed to identify cHL/PTCL patients treated with BV and retreated with BV-M, BV-C, or non-BV therapy. Patient characteristics were described by retreatment, and predictors of BV-M retreatment were identified. Among the cHL and PTCL patients treated with BV (n = 6442 and 2472, respectively), 13% and 12%, respectively, were retreated with BV; the median times from initial BV to BV-M retreatment were 5 and 7 months, respectively; and the numbers of BV-M retreatment doses were 4 and 5, respectively. Among cHL patients, the predictors of BV-M retreatment were age (18-39 vs. ≥60 years), sex (women vs. men), and previous stem cell transplantation (yes vs. no). Among PTCL patients, the only predictor of BV-M retreatment was systemic anaplastic large-cell lymphoma subtype (yes vs. no). Real-world data support clinical study results suggesting earlier BV treatment be considered, as BV retreatment may be an option.

Real-world use of inhaled corticosteroid/formoterol as-needed in adults with mild asthma: The PRIME study

IntroductionInhaled corticosteroid/formoterol fumarate (ICS/FF) as-needed is recommended by the Global Initiative for Asthma as sole therapy in adults with mild asthma, with low-dose maintenance ICS plus short-acting β2-agonist (SABA) as an alternative. SABA alone is no longer recommended. Given these changes in recommendations, the observational PRIME study aimed to describe real-world treatment patterns in mild asthma in Europe.MethodsAdults with asthma receiving low-dose maintenance ICS, or as-needed ICS/FF or SABA were followed for 6 months. Data collected included Asthma Control Test (ACT), Asthma Control Questionnaire 5 item (ACQ-5), forced expiratory volume in 1 s (FEV1), and asthma exacerbations.ResultsThe study was conducted in Germany, Italy, Poland and Spain, in 883 patients; 833 (94.3%) completed follow-up. At enrolment, 32.2% received maintenance ICS, 56.3% ICS/FF as-needed, and 11.6% SABA as-needed; 57.4%, 61.3%, and 54.9%, respectively, had well controlled asthma (ACQ-5/ACT definition). After 6 months, changes in mean FEV1were small in the maintenance ICS and ICS/FF as-needed groups, whereas there was a decline in FEV1in the SABA as-needed group. ACQ-5 total score improved from baseline in all three groups; 0.4%, 0.4% and 2.0% patients, respectively, had a severe exacerbation during the study.ConclusionsMore patients received ICS/FF as-needed than SABA as-needed, suggesting that physicians are aware of the latest treatment recommendations. This real-world study provides additional support to the use of ICS/FF as-needed as preferred treatment for patients with mild asthma, whereas SABA as-needed was associated with a fall in lung function and more severe exacerbations.

Real-World Treatment Patterns and Outcomes by Line of Therapy and Race in Patients With Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma Treated in the United States: Results From the Final Analysis of the Prospective, Observational, informCLL Registry

BackgroundinformCLL is the largest US-based prospective, observational registry of patients with chronic lymphocytic leukemia (CLL) initiating FDA-approved treatment in the era of targeted therapy. Patients and MethodsPatients were enrolled between October 2015 and June 2019. Data were collected for baseline characteristics, treatment patterns, outcomes, and safety. ResultsIn total, 1459 eligible patients were enrolled (first line, n = 854; relapsed/refractory, n = 605). The most common index treatments were ibrutinib (first line, 45%; relapsed/refractory, 49%) and chemoimmunotherapy (first line, 43%; relapsed/refractory, 20%). With median follow-up of 31.8 and 30.9 months in first-line and relapsed/refractory cohorts, respectively, median time to next treatment (TTNT) in patients who received any index treatment was not reached (NR) and 48.6 months; estimated proportions without next-line therapy at 48 months were 64% and 50%. Median overall survival (OS) was NR for both cohorts; estimated 48-month OS rates were 81% and 64% in first-line and relapsed/refractory cohorts, respectively. In match-adjusted analyses, TTNT was improved with first-line ibrutinib versus chemoimmunotherapy (median NR vs. 56.5 months; hazard ratio, 0.74; 95% CI, 0.56-0.98). Exposure-adjusted rates of AEs leading to discontinuation and serious AEs were lower with ibrutinib versus chemoimmunotherapy. Estimated 36-month OS rates were similar in Black versus White patients who received any index treatment (first line, 87% vs. 83%; relapsed/refractory, 74% vs. 74%) or ibrutinib (first line, 97% vs. 85%; relapsed/refractory, 81% vs. 77%). ConclusionIn this prospective, large, real-world CLL registry, first-line ibrutinib was associated with longer TTNT than chemoimmunotherapy, with sustained benefit up to 4 years of follow-up.

Real-world utilization, patient characteristics, and treatment patterns among men with localized prostate cancer tested with the 17-gene genomic prostate score® (GPSTM) assay.

Descriptive study focusing on real-world utilization and characteristics of men with prostate cancer tested with the 17-gene Genomic Prostate Score® (GPS™) assay by linking administrative claims and electronic health record (EHR) data with GPS results. This retrospective, observational cohort study (January 1, 2013 to December 31, 2020) included men aged 40-80 years with localized prostate cancer claims, continuous enrollment in Optum's Integrated Claims data set, ≥1 day of EHR clinical activity, and a GPS result. Men were classified as undergoing definitive therapy (DT) (prostatectomy, radiation, or focal therapy) or active surveillance (AS). AS and DT distribution were analyzed across GPS results, National Comprehensive Cancer Network® (NCCN®) risk, and race. Costs were assessed 6 months after the first GPS result (index); clinical outcomes and AS persistence were assessed during the variable follow-up. All variables were analyzed descriptively. Of 834 men, 650 (77.9%) underwent AS and 184 (22.1%) DT. Most men had Quan-Charlson comorbidity scores of 1-2 and a tumor stage of T1c (index). The most common Gleason patterns were 3 + 3 (79.6%) (AS cohort) and 3 + 4 (55.9%) (DT cohort). The mean (standard deviation) GPS results at index were 23.2 (11.3) (AS) and 30.9 (12.9) (DT). AS decreased with increasing GPS result and NCCN risk. Differences between races were minimal. Total costs were substantially higher in the DT cohort. Most men with GPS-tested localized prostate cancer underwent AS, indicating the GPS result can inform clinical management. Decreasing AS with increasing GPS result and NCCN risk suggests the GPS complements NCCN risk stratification.

Real-World Treatment Patterns and Survival Outcomes for Patients with Non-Metastatic Non-Small-Cell Lung Cancer in Sweden: A Nationwide Registry Analysis from the I-O Optimise Initiative.

Non-small-cell lung cancer (NSCLC) is the leading cause of cancer-related death worldwide, with ~40-50% of patients diagnosed with non-metastatic disease (stages IA-IIIC). The treatment landscape is evolving rapidly as immunotherapies and targeted therapy are introduced in the non-metastatic setting, creating a need to assess patient outcomes prior to their introduction. This real-world study using Swedish National Lung Cancer Registry data examined outcomes (overall survival (OS) and time to next treatment or death (TTNTD)) and treatment patterns for adults diagnosed with non-metastatic NSCLC. Baseline characteristics and OS from diagnosis were described for all patients; OS, treatment patterns, and TTNTD from treatment start were described for the treatment subgroup (patients diagnosed from 2014 onwards), stratified by disease stage and initial treatment. OS and TTNTD were described using the Kaplan-Meier estimator. The overall population (2008-2019) included 17,433 patients; the treatment subgroup included 5147 patients. Median OS (interquartile range) overall ranged from 83.3 (31.6-165.3) months (stage I patients) to 10.4 (4.3-24.2) months (stage IIIB patients). Among the treatment subgroup, median OS and TTNTD were longest among patients receiving surgery versus other anticancer treatments. These findings provide a baseline upon which to evaluate the epidemiology of non-metastatic NSCLC as newer treatments are introduced.

Real-world treatment patterns and medical costs of prostate cancer patients in Switzerland – A claims data analysis

BackgroundProstate cancer (PC) is the most prevalent cancer in men in Switzerland. However, evidence on the real-world health care use of PC patients is scarce. The aim of this study is to describe health care utilization, treatment patterns, and medical costs in PC patients over a period of five years (2014–2018). MethodWe used routinely collected longitudinal individual-level claims data from a major provider of mandatory health insurance in Switzerland. Due to the lack of diagnostic coding in the claims data, we identified treated PC patients based on the treatments received. We described health care utilization and treatment pathways for patients with localized and metastatic PC. Costs were calculated from a health care system perspective. ResultsA total of 5591 PC patients met the inclusion criteria. Between 2014 and 2018, 1741 patients had outpatient radiotherapy for localized or metastatic PC and 1579 patients underwent radical prostatectomy. 3502 patients had an androgen deprivation therapy (ADT). 9.5% of these patients had a combination therapy with docetaxel, and 11.0% had a combination with abiraterone acetate. Docetaxel was the most commonly used chemotherapy (first-line; n = 413, 78.4% of all patients in chemotherapy). Total medical costs of PC in Switzerland were estimated at CHF 347 m (95% CI 323–372) in 2018. ConclusionMost PC patients in this study were identified based on the use of ADT. Medical costs of PC in Switzerland amounted to 0.45% of total health care spending in 2018. Treatment of metastatic PC accounted for about two thirds of spending.

Treatment patterns in women with postmenopausal osteoporosis using abaloparatide: a real-world observational study.

Abaloparatide is approved for the treatment of women with postmenopausal osteoporosis at high risk for fracture. This study evaluated real-world treatment patterns for patients new to abaloparatide, regardless of osteoporosis treatment history. Data for patients with ≥ 1 prescription for abaloparatide were collected retrospectively from six academic and clinical practice settings across the US. A total of 173 patients were enrolled (mean [SD] age, 69.8 [7.4] years). At the time of abaloparatide treatment initiation, 78.6% had received other osteoporosis medications. Mean (SD) time from discontinuation of osteoporosis medications prior to initiation of abaloparatide was 1.7 (3.2) years. Twenty-four months of follow-up data from the initiation date of abaloparatide was collected from 94.0% of patients and 6.0% of patients had 12-24months of follow-up. During the follow-up period, 96.0% of patients had at least one visit for osteoporosis management and 55.5% had access to a medication support program. The median duration of therapy was 18.6months and 105/162 (64.8%) completed abaloparatide treatment as prescribed. The most common reasons for treatment discontinuation were financial (31.2%) and tolerability (22.8%). Following completion of a course of treatment with abaloparatide, 82/162 (50.6%) patients transitioned to another osteoporosis medication. The median time between abaloparatide treatment course completion and the initiation of follow-on medication was 21days. Most patients completed treatment with abaloparatide as prescribed, and over half continued with an antiresorptive agent. This favorable conduct may be the result of regular follow-up visits and accessibility to both medication and patient support services.

Real-world progression-free survival and overall survival of palbociclib plus endocrine therapy (ET) in Japanese patients with hormone receptor-positive/human epidermal growth factor receptor 2-negative advanced breast cancer in the first-line or second-line setting: an observational study.

A recent large real-world study conducted in the United States reported the effectiveness of palbociclib plus aromatase inhibitor in HR+/HER2- advanced breast cancer (ABC). However, local clinical practice and available medical treatment can vary between Japan and Western countries. Thus, it is important to investigate Japanese real-world data. This observational, multicenter study (NCT05399329) reports the interim analysis of effectiveness of palbociclib plus ET as first-line or second-line treatment for HR+/HER2- ABC by estimating real-world progression-free survival (rwPFS) and overall survival (OS) in Japanese routine clinical practice. Real-world clinical outcomes and treatment patterns of palbociclib plus ET were captured using a medical record review of patients diagnosed with HR+/HER2- ABC who had received palbociclib plus ET in the first-line or second-line treatment across 20 sites in Japan. The primary endpoint was rwPFS; secondary endpoints were OS, real-world overall response rate, real-world clinical benefit rate, and chemotherapy-free survival. Of the 677 eligible patients, 420 and 257 patients, respectively, had received palbociclib with ET as first-line and second-line treatments. Median rwPFS (95% confidence interval) was 24.5months (19.9-29.4) for first-line and 14.5months (10.2-19.0) for second-line treatment groups. Median OS was not reached in the first-line group and was 46.7months (38.8-not estimated) for the second-line group. The 36-month OS rates for de novo metastasis, treatment-free interval (TFI) ≥ 12months, and TFI < 12months were 80.2% (69.1-87.7), 82.0% (70.7-89.3), and 66.0% (57.9-72.9), respectively. The addition of palbociclib to ET was effective for treating HR+/HER2- ABC in Japanese routine clinical practice.

Real-World Treatment Patterns and Healthcare Resource Use for Ulcerative Colitis and Crohn's Disease in Italy.

Real-world data are used to inform decision-makers and optimise therapeutic management for patients with ulcerative colitis (UC) and Crohn's disease (CD). We analysed data on the epidemiology (by using proxies of prevalence and incidence), patient characteristics, treatment patterns and associated healthcare direct costs for the management of patients with UC and patients with CD in Italy. This retrospective observational study used administrative databases from eight Local Health Units geographically distributed across Italy. Adult patients with a hospitalisation and/or an exemption for UC or CD were included. Study outcomes were summarised descriptively, and limited statistical tests were performed. At baseline, 9255 adults with UC and 4747 adults with CD were included. Mean (standard deviation) age at inclusion was 54.0 (18.4)/48.6 (18.1) years, for UC/CD. The estimated average incidence of UC and CD for the period2013-2020 was 36.5 and 18.7 per 100,000, respectively. The most frequently prescribed drug category for patients with UC/CD was conventional treatment [mesalazine and topical corticosteroids (67.4%/61.1%), immunomodulators and systemic corticosteroids (43.2%/47.7%)], followed by biologic treatments (2.1%/5.1%). The mean annual total direct cost per patient was 7678euro (€), for UC and €6925 for CD. This analysis, carried-out in an Italian clinical setting, may help to optimise therapy for patients with UC and CD and provide relevant clinical practice data to inform decision-makers.

Tafamidis 61mg Patient Characteristics and Persistency? A Retrospective Analysis of German Statutory Health Insurance Data (IQVIA™ LRx).

Tafamidis is the first drug approved by the European Commission for the treatment of wild-type or hereditary transthyretin amyloid cardiomyopathy (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. Real-world treatment patterns of tafamidis 61mg in Germany are not well studied in patients with ATTR-CM. This was a non-interventional, retrospective, observational cohort study of adult patients in Germany based on the IQVIA pharmacy claims database (IQVIA™ LRx). Patients included in the analysis were statutory insured and received at least one prescription of tafamidis 61mg between March 1, 2020 and August 31, 2022. Treatment adherence was analyzed using the modified medical possession ratio (mMPR) and proportion of days covered (PDC). Overall, 1565 adult patients received at least one tafamidis prescription in the study period. Their mean age was 78.3years, 82.4% were male, and 23.2% were treated by a cardiologist. Persistency rates for patients treated with tafamidis 61mg were high: 78.0% for 12months and 65.1% for 24months after treatment initiation. Patients also had high adherence rate on filling their prescriptions on time: 94.6% and 90.5% of patients had adherence rates of at least 80%, measured by mMPR and PDC, respectively. In the IQVIA™ LRx database, patients prescribed tafamidis 61mg in Germany displayed high adherence and persistency rates, which suggest good drug tolerability and ease of use.

Real-World Treatment Patterns and Clinical Outcomes among Patients Receiving CDK4/6 Inhibitors for Metastatic Breast Cancer in a Canadian Setting Using AI-Extracted Data.

Cyclin-dependent kinase 4/6 inhibitors (CDK4/6i) are widely used in patients with hormone receptor-positive (HR+)/human epidermal growth factor receptor 2 negative (HER2-) advanced/metastatic breast cancer (ABC/MBC) in first line (1L), but little is known about their real-world use and clinical outcomes long-term, in Canada. This study used Pentavere's previously validated artificial intelligence (AI) to extract real-world data on the treatment patterns and outcomes of patients receiving CDK4/6i+endocrine therapy (ET) for HR+/HER2- ABC/MBC at Sinai Health in Toronto, Canada. Between 1 January 2016 and 1 July 2021, 48 patients were diagnosed with HR+/HER2- ABC/MBC and received CDK4/6i + ET. A total of 38 out of 48 patients received CDK4/6i + ET in 1L, of which 34 of the 38 (89.5%) received palbociclib + ET. In 2L, 12 of the 21 (57.1%) patients received CDK4/6i + ET, of which 58.3% received abemaciclib. In 3L, most patients received chemotherapy (10/12, 83.3%). For the patients receiving CDK4/6i in 1L, the median (95% CI) time to the next treatment was 42.3 (41.2, NA) months. The median (95% CI) time to chemotherapy was 46.5 (41.4, NA) months. The two-year overall survival (95% CI) was 97.4% (92.4, 100.0), and the median (range) follow-up was 28.7 (3.4-67.6) months. Despite the limitations inherent in real-world studies and a limited number of patients, these AI-extracted data complement previous studies, demonstrating the effectiveness of CDK4/6i + ET in the Canadian real-world 1L, with most patients receiving palbociclib as CDK4/6i in 1L.

Real World Treatment Patterns of Patients With Dravet Syndrome (DS) and Lennox-Gastaut Syndrome (LGS) in the United States (US) (P1-1.005)

Real World Treatment Patterns of Patients With Dravet Syndrome (DS) and Lennox-Gastaut Syndrome (LGS) in the United States (US) (P1-1.005)

Patterns of care and costs of switching oral antipsychotic medications in patients with schizophrenia initiating monotherapy treatment: A US claims analysis.

Antipsychotic medications are the mainstay of schizophrenia therapy but may need to be changed over the course of a patient's illness to achieve the desired therapeutic goals or minimize medication side effects. Investigations of real-world treatment patterns and economic consequences associated with antipsychotic changes, including switching, are limited. To describe treatment patterns among patients with schizophrenia who initiated oral antipsychotic medication (OAM) monotherapy and assess switching-related health care resource utilization (HCRU) and costs in US Medicare Advantage and commercially insured patients. Adults with at least 2 claims with a schizophrenia diagnosis who initiated (or reinitiated after ≥6 months) OAM monotherapy between January 1, 2015, and June 30, 2021, were identified in the Optum Research Database. A claims-based algorithm using timing of therapies and treatment gaps identified medication changes, specifically OAM monotherapy switches, among OAM initiators over a period of up to 7 years. Patients who switched from their initial OAM monotherapy to a second OAM monotherapy (initial OAM switchers) were matched based on clinical and demographic characteristics to OAM initiators who had not switched OAMs; switch-related HCRU and costs (incurred up to 3 months before and 3 months after the initial OAM switch) were compared between matched initial OAM switchers and nonswitchers. Among 6,425 OAM monotherapy initiators, 1,505 (23.4%) had at least 1 OAM monotherapy switch at any time during follow-up, with a mean (SD) time to first switch of 209 (333) days (median, 67 days), a rate of 0.65 switches per person-year of follow-up, and 56% of first switches occurring within 3 months of OAM initiation. Of all OAM initiators, 947 (14.7%) were initial OAM switchers. Compared with 865 matched nonswitchers, 865 initial OAM switchers had greater mean counts of all-cause medical visits and greater mean counts of schizophrenia-related emergency and inpatient visits and longer inpatient stays per patient per month. Mean (SD) total schizophrenia-related costs per patient per month were $1,252 ($2,602) for switchers compared with $402 ($2,027) for nonswitchers (P < 0.001). Changes to antipsychotic therapy in our sample of patients with schizophrenia were common, with nearly one-fourth switching OAMs, the majority within the first 3 months of therapy. Initial OAM switchers experienced greater HCRU and costs than nonswitchers. These findings highlight the importance of initiating OAM monotherapy that effectively maintains symptom control and minimizes tolerability issues, which would limit the need to switch OAMs and therefore prevent excess HCRU and treatment costs.